Global Harmonization of Pediatric Drug Development: Critical for Progress for Developing Safe and Effective Therapeutic Agents for Children

por Mulberg, Andrew E., Cripps, Timothy
Publicado 2019

Coefficient of Fat Absorption to Measure the Efficacy of Pancreatic Enzyme Replacement Therapy in People With Cystic Fibrosis: Gold Standard or Coal Standard?

por Borowitz, Drucy, Aronoff, Nell, Cummings, Linda C., Maqbool, Asim, Mulberg, Andrew E.
Publicado 2022

Sharing is caring: a call for a new era of rare disease research and development

por Denton, Nathan, Mulberg, Andrew E., Molloy, Monique, Charleston, Samantha, Fajgenbaum, David C., Marsh, Eric D., Howard, Paul
Publicado 2022

Correction to: Sharing is caring: a call for a new era of rare disease research and development

por Denton, Nathan, Mulberg, Andrew E., Molloy, Monique, Charleston, Samantha, Fajgenbaum, David C., Marsh, Eric D., Howard, Paul
Publicado 2023

Data silos are undermining drug development and failing rare disease patients

por Denton, Nathan, Molloy, Monique, Charleston, Samantha, Lipset, Craig, Hirsch, Jonathan, Mulberg, Andrew E., Howard, Paul, Marsh, Eric D.
Publicado 2021

FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument

por Shields, Alan L., Lamoureux, Roger E., Taylor, Fiona, Barth, Jay A., Mulberg, Andrew E., Kessler, Vivian, Skuban, Nina
Publicado 2021

Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity

por Harfouche, Melodi, Kishnani, Priya S., Krusinska, Eva, Gault, Jamie, Sitaraman, Sheela, Sowinski, Amanda, Katz, Irina, Austin, Stephanie, Goldstein, Margi, Mulberg, Andrew E.
Publicado 2020

Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders

por Mulberg, Andrew E., Bucci-Rechtweg, Christina, Giuliano, Joseph, Jacoby, David, Johnson, Franklin K., Liu, Qing, Marsden, Deborah, McGoohan, Scott, Nelson, Robert, Patel, Nita, Romero, Klaus, Sinha, Vikram, Sitaraman, Sheela, Spaltro, John, Kessler, Vivian
Publicado 2019

Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial

por Schiffmann, Raphael, Bichet, Daniel G., Jovanovic, Ana, Hughes, Derralynn A., Giugliani, Roberto, Feldt-Rasmussen, Ulla, Shankar, Suma P., Barisoni, Laura, Colvin, Robert B., Jennette, J. Charles, Holdbrook, Fred, Mulberg, Andrew, Castelli, Jeffrey P., Skuban, Nina, Barth, Jay A., Nicholls, Kathleen
Publicado 2018